HUMAN GENE THERAPY
Human Gene Therapy is the official journal of the European Society for Gene and Cellular Therapy. A rapid-publication peer-reviewed journal covering all aspects of human gene therapy. Publishes scientific papers on original investigations into the transfer and expression of genes in mammals, including humans. Improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, are covered. Includes ethical/legal/regulatory papers related directly to the area of gene transfer into humans.
 Human Gene Therapy - Table of Contents Selective Modification of Antigen-Specific T Cells by RNA Electroporation Human Gene Therapy , Vol. 0, No. 0: 1-11. It has been observed that the efficient transfection of T cells by RNA electroporation requires prior activation of T cells with mitogens or by anti-CD3 antibody stimulation. We hypothesized that this requirement for T cell activation could be leveraged ... Author : liebertonline@liebertpub.com (Duane A. Mitchell et al) Modified Concatemeric Oligonucleotide Complexes: New System for Efficient Oligonucleotide Transfer into Mammalian Cells Human Gene Therapy , Vol. 0, No. 0: 1-16. Antisense oligonucleotides and double-stranded small interfering RNAs have become an important instrument for the manipulation of gene expression in molecular biology experiments and a promising tool for the development of gene-targeted therapeutics. One ... Author : liebertonline@liebertpub.com (Olesya N. Gusachenko (Simonova) et al) Interaction of Systemically Delivered Adenovirus Vectors with Kupffer Cells in Mouse Liver Human Gene Therapy , Vol. 0, No. 0: 1-8. When adenovirus (Ad) vectors are injected intravenously they are rapidly taken up by Kupffer cells (KCs) in the liver. This results in massive KC necrosis within minutes, followed by a more gradual disappearance of KCs from the liver. It is not known how ... Author : liebertonline@liebertpub.com (Jeffrey S. Smith et al) Adoptive Cell Therapy for Patients with Melanoma, Using Tumor-Infiltrating Lymphocytes Genetically Engineered to Secrete Interleukin-2 Human Gene Therapy , Vol. 0, No. 0: 1-16. Adoptive cell transfer of tumor-infiltrating lymphocytes (TILs) after lymphodepletion mediates regression in 50% of patients with metastatic melanoma. In vivo persistence and telomere length of the transferred cells correlate with antitumor response. In ... Author : liebertonline@liebertpub.com (Bianca Heemskerk et al) The Business of RNAi Therapeutics Human Gene Therapy , Vol. 0, No. 0: 1-12. Author : liebertonline@liebertpub.com (Dirk Haussecker) Characterization of Complete Particles (VSV-G/SIN-GFP) and Empty Particles (VSV-G/EMPTY) in Human Immunodeficiency Virus Type 1-Based Lentiviral Products for Gene Therapy: Potential Applications for Improvement of Product Quality and Safety Human Gene Therapy , Vol. 0, No. 0: 1-12. Lentiviral vectors persist in the host and are therefore ideally suited for long-term gene therapy. To advance the use of lentiviral vectors in humans, improvement of their production, purification, and characterization has become increasingly important ... Author : liebertonline@liebertpub.com (Yuan Zhao et al) Expression Strategies for Short Hairpin RNA Interference Triggers Human Gene Therapy Apr 2008, Vol. 19, No. 4: 313-317. Since the discovery that the triggers for RNA interference (RNAi), small interfering RNAs, could mediate silencing in mammalian cells without triggering a toxic response, RNAi has become the standard tool for sequence-specific knockdown of gene ... Author : liebertonline@liebertpub.com (John J. Rossi) Delivery of Recombinant Adeno-Associated Virus-Mediated Human Tissue Kallikrein for Therapy of Chronic Renal Failure in Rats Human Gene Therapy Apr 2008, Vol. 19, No. 4: 318-330. The tissue kallikrein–kinin system is important in regulating cardiovascular and renal function, and dysregulation of the system has been implicated in heart and kidney pathologies. These findings suggest that if balance can be restored to the kallikrein–... Author : liebertonline@liebertpub.com (Ling Tu et al) Therapy of Anemia in Kidney Failure, Using Plasmid Encoding Erythropoietin Human Gene Therapy Apr 2008, Vol. 19, No. 4: 331-342. Numerous studies using erythropoietin (EPO) gene delivery vectors, either viral or nonviral, have shown uncontrolled EPO expression leading to transient or sustained erythrocytosis and, more recently, severe autoimmune anemia. Therefore, there is a need ... Author : liebertonline@liebertpub.com (Peggy Richard-Fiardo et al) Potent Antitumor Efficacy of ST13 for Colorectal Cancer Mediated by Oncolytic Adenovirus via Mitochondrial Apoptotic Cell Death Human Gene Therapy Apr 2008, Vol. 19, No. 4: 343-353. ST13 is a cofactor of heat shock protein 70 (Hsp70). To date, all data since the discovery of ST13 in 1993 until more recent studies in 2007 have proved that ST13 is downregulated in tumors and it was proposed to be a tumor suppressor gene, but no work ... Author : liebertonline@liebertpub.com (Min Yang et al) Antitumor Activity of DNA Vaccines Based on the Human Papillomavirus-16 E7 Protein Genetically Fused to a Plant Virus Coat Protein Human Gene Therapy Apr 2008, Vol. 19, No. 4: 354-364. DNA vaccination represents an attractive strategy for cancer immunotherapy combining vaccine stability, cost-effectiveness, and safety. However, a major problem of genetic vaccination is the limited potency, due to intrinsic lack of amplifying and ... Author : liebertonline@liebertpub.com (Silvia Massa et al) Optimization of Recombinant Adeno-Associated Viral Vectors for Human β-Globin Gene Transfer and Transgene Expression Human Gene Therapy Apr 2008, Vol. 19, No. 4: 365-375. Therapeutic levels of expression of the β-globin gene have been difficult to achieve with conventional retroviral vectors without the inclusion of DNase I-hypersensitive site (HS2, HS3, and HS4) enhancer elements. We generated recombinant adeno-... Author : liebertonline@liebertpub.com (Njeri Maina et al) Recombinant Self-Complementary Adeno-Associated Virus Serotype Vector-Mediated Hematopoietic Stem Cell Transduction and Lineage-Restricted, Long-Term Transgene Expression in a Murine Serial Bone Marrow Transplantation Model Human Gene Therapy Apr 2008, Vol. 19, No. 4: 376-383. Although conventional recombinant single-stranded adeno-associated virus serotype 2 (ssAAV2) vectors have been shown to efficiently transduce numerous cells and tissues such as brain and muscle, their ability to transduce primary hematopoietic stem cells ... Author : liebertonline@liebertpub.com (Njeri Maina et al) Noninvasive In Vivo Delivery of Transgene via Adeno-Associated Virus into Supporting Cells of the Neonatal Mouse Cochlea Human Gene Therapy Apr 2008, Vol. 19, No. 4: 384-390. There are a number of genetic diseases that affect the cochlea early in life, which require normal gene transfer in the early developmental stage to prevent deafness. The delivery of adenovirus (AdV) and adeno-associated virus (AAV) was investigated to ... Author : liebertonline@liebertpub.com (Takashi Iizuka et al) Gene Gun Bombardment with DNA-Coated Gold Particles Is a Potential Alternative to Hydrodynamics-Based Transfection for Delivering Genes into Superficial Hepatocytes Human Gene Therapy Apr 2008, Vol. 19, No. 4: 391-395. Although in vivo nonviral gene delivery to the liver is critical for hepatic gene therapy, there are a number of technical obstacles. Enhanced green fluorescent protein (EGFP)-encoding DNA was coated onto gold particles (gold–DNA), dissolved in phosphate-... Author : liebertonline@liebertpub.com (Ming-Ling Chang et al) Increased Web Site Traffic |
